Watch the videos from the UK MDS Forum Education Day 2016

The following videos come from the annual UK MDS Forum Education Day 2016, which took place in London on 11th November 2016.

The day is targeted at all haematologists in the UK and Ireland who deal with MDS patients. Its aim is to provide information and updates on the most advanced scientific and clinical research in MDS. Please have a look at the agenda here.

We are now able to make the videos available online. Although these were initially intended for health professionals, patients and their families are also invited to watch. Our aim is to make this valuable information more widely available, not only to clinicians and nurses but also to patients, in order to educate and empower them, as well as explaining the fantastic research that goes on in MDS.

Since NHS England initially announced in December 2016 that it wouldn’t fund second transplants, more than 25,000 people joined our campaign to reverse the decision.

Thanks to every single person that signed the petitions, and wrote to their MPs. We all did it together.

Thanks to colleagues at Anthony Nolan, all other blood cancer charities, Emma Paine, Mark Tami MP, the APPG on Stem Cell Transplant, many MPs and clinicians, Sasha Jones and friends.

An extra-special thanks to the Hepburn family, who showed so much courage and campaigned selflessly after Gavin's death. We owe them a huge debt of gratitude.

NHS Announcement in detail: routine funding for 2nd stem cell transplants for patients who relapse more than a year after their 1st transplant

On Friday 24 February NHS England announced that they will routinely fund second stem cell transplants for patients who relapse more than one year after their first transplant. This replaces the announcement in December 2016 that second stem cell transplants were ‘not currently affordable’.

Every year, a small number of patients with a blood cancer or blood disorder who have already received one stem cell transplant from a donor will unfortunately relapse (their disease will come back). For some of these patients, their doctor might recommend a second donor stem cell transplant. It is estimated that 16 to 20 people every year in England will need a second transplant because their blood cancer or blood disorder has relapsed.

This decision affects a small number of patients in England who:
• have received a first donor (allogenic) stem cell transplant;
• were in complete remission;
• subsequently relapsed more than 12 months after their first transplant; and
• their doctor now recommends a second stem cell transplant.

This decision does not affect patients who suffer graft failure (their first transplant fails) or patients who have received a first transplant using their own cells (autologous). This decision only affects patients in England; patients in other parts of the UK are not affected.

Why does this decision only apply to patients who relapsed more than 12 months after their first transplant?

The recommendation of clinical experts is that patients who relapse more than 12 months after their first transplant have the best chance of a successful second transplant. Unfortunately, there is weak evidence for the effectiveness of second transplants for patients who relapse within a year of their first donor transplant.

Read More: Blood and Marrow Transplantation

More information on 2nd stem cell transplants on the NHS official site:

Early in 2016 a MDS patient was denied a potentially life- saving 2nd bone marrow transplant despite showing a good chance of being cured by this 2nd treatment.

After 4 months of waiting, spent in and out of hospital, while NHS England turned down the treatment again, following an appeal, this patient contracted a severe infection and died.

He was only 39 years old – and a father of 3 young children.

End of 2016:

A mum with 2 young children is finding herself in a similar situation. She needs a 2nd transplant after a relapse of her blood cancer, Acute Myeloid Leukaemia.

The request for funding was denied just recently – meaning she has 2 choices: find the £100k herself to pay for the transplant or accept she may only have months left to live.

What is the background behind these appalling situations?

At the end of 2013, NHS England took the decision to stop funding allogeneic stem cell transplants (allo HSCT) for blood cancer patients who suffered a relapsed of their disease – after an initial successful transplant.
Expert clinicians requested a review of this decision, which did happen.

But in July 2016 NHS England came to a decision and stated they would no longer routinely fund 2nd allo HSCT’s – based on the evidence and success rates. These treatments were no longer deemed to be “cost-effective” for the current financial situation of the NHS.

It is true that overall, outcomes of 2nd transplants are not great – on average.
But transplants, types of blood cancer, sub-types, patients are very unique and these situations are rarely comparable.
Not only is the need for a 2nd HSCT rare, but not many patients would be so eager to undergo a 2nd transplant. Nor would haematologists be willing to perform one if there was not some good chance it would save someone’s life.
Clinicians and patients understand the issue of quantity versus quality of life.

Ever since 2013, haematologists deciding their patients would benefit from a 2nd HSCT, would have to make a compelling case for it – via an IFR (Individual Funding Request) which would then be submitted to an NHS England IFR Panel.
The IFR panel would then discuss whether the case was exceptional enough to agree the funding.

What does that mean in practice and in actual figures?¹

A bone marrow transplant costs about £100k per patient on average.
Between April 2013 and June 2016, only 19 requests for a 2 nd HSCT were made in all of England:

5 were declined by the IFR Screening team (the case was never seen by the IFR Panel)5 were declined by the IFR panel3 had “other outcome” (sic - as per NHS FOI answer)6 were approved – this means just 2 cases per year. Total cost of approx £200k.

The 2 recent patients mentioned have been denied this chance – to save NHS England £200k.

It is crystal clear that the NHS cannot afford to treat everyone whatever the cost – so let’s set aside the
emotional and humanity argument.

It is the money side and management of NHS funds we take issue with.

NHS England refuses to consider, or key budget holders seem unable to take into account the cost of keeping these patients alive with supportive care – or palliative care.
The patient who died ended up costing the NHS closer to £250k – just to keep him alive for 5-6 months before his death – between frequent hospital stays, tons of antibiotics, expensive new medication, frequent complex blood and platelet transfusions.

A saving of £100k = an NHS bill of £250k + a deathThis is not tax payer’s money well spent

Furthermore – we have uncovered that despite NHS England’s explanations, IFR cases are not assessed by transplant specialists, but by a mix of “trained” IFR staff, pharmacists and public health consultants or another random types of physicians.
There is no process forcing IFR staff to submit highly complex transplant IFR’s to relevant experts in that field.
There is an ongoing NHS England consultation on this very topic closing on 15/01/17.

Anthony Nolan, together with the Blood Cancer Alliance community and leading experts, published an open letter to the Secretary of State for Health (Times, July 2016) and led a significant campaign – but to no avail:

“We believe this is simply wrong. If a stem cell transplant is the best option for a patient, and a donor can be found, we believe the treatment should be available. We know that there are many people alive today, leading fulfilling and active lives with their families, because they had a second transplant.”
(Anthony Nolan campaign extract).

¹ Figures obtained through Freedom of Information Act enquiries – and from medical notes shared by the family

Below are the full stories of both families.

This makes for very difficult reading.

We urge the government to review this nonsensical and inhumane situation.

There are cases where patients do stand a good chance to have a successful 2nd transplant – but these decisions must be made by expert haematologists – not public health physicians, let alone non-clinical staff – however well trained.

The savings are a false economy, as evidenced in this case.Supportive care ends up costing twice as much, patients occupy badly needed hospital beds, families lose a bread-winner and government a tax-payer.

I am a fun loving 34 year old nail artist from Greenwich, London. I am the wife of my childhood sweetheart Lloyd Williams and Mother to our 13 year old daughter Katia and 8 year old Son Lj Williams. I am fighting for a second bone marrow transplant. I am Sasha Jones (Williams) and I deserve to be alive just like you.

After a routine blood test on the morning of March 11 th 2015 due to me feeling unwell, I was sadly given the devastating news that the tests suggested I was suffering from Acute Myeloid Leukaemia and was admitted within hours.

On Sunday 15 th March I started my chemotherapy treatment and a bone marrow aspirate revealed that the only chance I had of keeping the disease at bay was to have a bone marrow transplant. So the search begun to find a donor and my treatment commenced.

After my first round of chemo it was revealed the amazing news that I was in complete remission and had 3 donor matches!
3 rounds of chemo over a 5 month period later and I was admitted to Kings College Hospital for my graft. On 26th August 2016 and after 1 week of high intensity chemotherapy treatment I was given the bone marrow transplant I so desperately needed in the hope my leukaemia would stay in remission.

After 18 days in hospital, lots of blood treatment, pain management and rest I was relieved to seem well enough to go home to my husband and children. However, this was short lived when I was re-admitted 3 days later after Kings discovering the activation of a virus passed on through my new donor. I then spent another grueling 14 days in hospital trying to get rid of the reactivated virus only to be released home again to start my recovery.

But, yet again after another 2 days at home I was crucifying readmitted once more for yet another 14 days due to another virus called BK 2000 causing haemorrhagic cystitis. This left me with months and months of grumbling bladder pain (which I still have too this very day every time I urinate) and incontinence which made the recovery process a very uncomfortable, embarrassing and painful process.

By the beginning of October I returned home and managed to then stay home with medication and 2 visits a week to Kings College Hospital to monitor my progress. On top of all this my donor and I did not have the same blood type which also caused problems up until as recent as August 2016 when my blood changed to a type A+ from my original O+.

During 2016 I visited the hospital on a weekly basis and it started to look like everything was going right.
My recovery was long and hard but I never let it get me down, I had to do it for the children!
I could see the light at the end of the tunnel with my 1 year bone marrow milestone approaching on 26th August 2016!
Sadly my happiness was short lived...

On Tuesday 9th August 2016 after a routine blood test again I got the devastating news that my new bone marrow was now producing leukaemia cells and that the transplant had failed.
This was confirmed by a bone marrow aspirate result on August 23rd 2016, I had relapsed, the dreaded cancer had returned and I needed to start my battle all over again beginning with chemotherapy followed by an infusion of donor cells once in remission.
I was to be put forward for new clinical trials using donor cells and a vaccine to give me any hope of surviving without a second transplant as this option had been stripped from patients due to NHS cut backs. NHS England had announced that it would not routinely fund second stem cell transplants for patients with blood cancer or blood disorders who have relapsed.

I was then hit by the news that not only was NHS England denying patients these life-saving 2nd transplants, but that also my donor was no longer available to offer an infusion of cells or to assist with the completion of the trial.

I have therefore been left with no alternative than to have a 2nd bone marrow transplant and I’m currently waiting on a decision from NHS England as to whether they are going to provide me with the funding I so desperately need to save my life.
Given that they cannot offer me an alternative end to my treatment as my donor has been removed from the register due to circumstances unbeknownst to me, it is believed that under these exceptional circumstances (seeing as my life itself isn’t important enough) that I SHOULD be entitled to another bone marrow transplant to give me a chance of fighting the disease and beating it with a better matched donor that I have waiting for me right now.

I am young, in the prime of my life, have everything going for me and everything to live for, not to mention my 2 children who deserve the right to grow up with their mother. They have also the right to the love, support, care and stability that only a mother’s love can give.
We as a society and nation try only too hard every day to promote and push - only to take that chance away from 2 children for a mere £120k by not handing their mum a life line that they have such easy access to......

Another 2 children’s lives do not need to be destroyed, cancer already takes so many of us.
Why are we now giving up on us....
When did we stop trying to save lives over money but at the same time we put money into research to save lives that you then later let die...

MDS UK update: Sasha has been refused the 2nd transplant by NHS England.

Thanks to your help her private fundraising has reached about £110k and the funding of stem-cell transplants was discussed in Parliament! This is a huge step in the right direction. Please keep sharing her story and sign the petitions to make Sacha the last patient that needs to pay for a 2nd transplant.

Family of Gavin – RIP

This is a summary of events – from diagnosis in 2012 – through to Gavin’s passing on the 6th May 2016.Written by Lisa, his wife.

Nov 2012: Gavin gets his MDS diagnosis. Devastated and confused we are told he will need a bone marrow transplant in the new year.

June 2013: Gavin receives his first transplant and is home in little over 2 weeks. We are all elated and pray this is the new beginning for us all.

Oct 2013: Gavin is taken back into Kings for an infection that is destroying his red blood cells. Extremely ill his life is quite literally in the balance. But he fights it and is home and well within 3 weeks.
In between the above and September 2014 Gavin has top up transfusions of red blood cells and platelets (but rarely at this point).
The doctors are pleased and allow us all to travel to the USA for a month’s holiday.
Gavin was the healthiest he had been in a long time and we had a wonderful time.

Sept 2014: Gavin becomes unwell. A Bone Marrow Biopsy suggests that he may have relapsed.
A course of Chemotherapy is advised. We are all devastated but remain positive.
Gavin's condition is very up and down between September and December and he is taken into Kings before Christmas with another infection.
The Rollercoaster that is MDS is truly devastating to us all.

2015: Not great - highs and lows, lots of infections and stays at Kings and more Chemo, that had to be stopped.

June 2015: Gavin is needing regular red blood cell and platelets transfusions. A planned holiday to Menorca is cancelled due to Gavin's condition. We head to Devon for 3 weeks, but the holiday is full of interruptions as Gavin needs regular transfusions at Plymouth Hospital. He remains positive though, but we are dreading the thought that another transplant would be needed.

November 2015: We receive the devastating news that Gavin will require another transplant. His condition at this point is very up and down with infections putting him back into Kings at regular intervals, again, in the run up to Christmas. At this point he is also living his life going backwards and forwards for transfusions at our local hospital twice a week. This is becoming very draining on him and us as a family. But we remain positive.

Feb 2016: We receive the devastating news that Gavin's funding for transplant has been refused.
How? Why?
We arrange a meeting with our MP Nicholas Soames. He is very sympathetic and agrees to bring up Gavin's case with parliament.
Ironically that evening an ambulance is called as Gavin is taken ill with another infection.
We vow to raise the money, but are advised that another application for transplant will be put forward.
We feel like we are not doing anything to get this ball rolling - this is his life and we are desperate.Gavin's condition at this point is up and down, and he is struggling to remain positive.

April 2016: After a wonderful day at the Isle of Wight, Gavin gets rushed back into hospital, East Surrey, our local hospital. It's the start of the 3 day doctor’s strike. We are all scared / devastated that this is happening again. On arrival there, we are told he will get the best care . How wrong were we. Gavin waits 8 hours for a simple bag of blood, he is climbing the walls in pain.
The doctors diagnose Pleurisy. He is put onto morphine for the pain. Gavin stays there for 3 days, and is not put onto the Kings Care plan until that 3rd day (when the doctors return back to work). At our total insistence as a family we get him transferred to Kings.
Dr Raj advises he needs this transplant but he needs to get well first. But he won't be leaving without it (hope again).
Gavin's condition deteriorates. He has a Lung Wash to clear the infection, the worst thing I've had to sit and listen too, I just wanted to run in and make them stop.
He improves a little and seems brighter. The morphine though has made him confused about things (very upsetting for myself and the children). It has also made him very constipated and blocked.
He can barely eat or drink.
It just feels like we are going 1 step forward 10 steps back.
The children just want their fun loving Daddy home and I my husband.

1st May 2016: Gavin gets told that there is nothing more they can do... we both can't believe it and won't believe it.
We put ourselves in a bubble and choose to ignore. Go to Costa and talk about anything but THAT.

2nd May 2016: Take the children to see him, very upsetting. He could barely be around them for no more than 15 mins.
Not like Gavin at all. He begs me not to leave that night.

Tuesday 3rd May 2016: I'm met by the palliative team at Kings. There is nothing more that can be done but to make him comfortable. I feel like my whole world has been blown up in front of me. My children, how do I tell them? We call all our family and friends. There is a constant flow of people for days.

Wednesday 4th May 2016: The children arrive and I tell them the news. They all react differently. Lots of tears, screaming the disbelief. I can safely say it was the hardest thing ever, but actually worst was to come.
We spend days and nights sitting and talking to him. The girls are amazing with him. Nursing him, holding his hand, cuddling him. Quite amazing for a 11 and 9 year old.

Friday 6th May 2016: My world is changed forever, our world. Gavin passes at 6.09am the exact time he was born on 17th Sept 1976. My mum brings the children up, I tell them there's another star in the sky.
They are destroyed - I am destroyed.

This should NEVER have happened. My gorgeous loving husband who had a zest for life and wanted to live was gone.
Myself and the children, 8 months on, are still devastated.
We receive regular counselling to get us through our grief, the pain and trying to understand WHY ?
The NHS failed Gavin when he needed it most...

Give Sasha a Chance!

Add your signature to Sacha's page on Change.org

A publicly funded NHS

The NHS must remain free & never be privatised

We are regularly in touch with the family – but Lisa and the children are still understandably struggling heavily with the loss of Gavin – and therefore all enquiries should be directed to MDS UK. Thank you for your understanding.We also sincerely thank the whole extended family, their friends, colleagues, employer for their ongoing support, participation, fundraising and awareness raising of MDS.We have the deepest of admiration for Gavin’s two daughters especially, who have displayed an incredible courage, maturity and strength over the last 3 years.They have spoken up about MDS, in school assembly, with friends, their athletics club, written to the press, participated in MDS UK activities, swam and baked for fundraisers, filmed an amazing awareness clip with their little brother – and even cut their hair off to make wigs for children with cancer.

There is hardly anyone they have not engaged around them- and still do so.

Please look up all of the above on our website www.mdspatientsupport.org.ukWe are immensely proud of them-and will continue to fight this issue in memory of Gavin – and for his family.Their wish now is simply that no family should ever need to go through what they are experiencing – this is why they agreed to share these details now.

Research FOR PatientsFor an informed and empowered opinionand an improved consultationHave you made your clinical paper accessible yet?

Bone Marrow Biopsies: a less painful alternative for routine check-ups

Until now, bone marrow sampling has been the primary technique for routine follow-up checks on MDS patients after initial diagnosis. The bone marrow is the heart of the disease and reveals important clues, for example, about whether a patient is responding to therapy or whether the disease is stable or worsening (progression).

During the procedure, which can be uncomfortable, an aspiration from the patient's marrow is taken, and specific blood cells derived from the bone marrow are analysed, allowing clinicians to monitor the ongoing disease status of a patient.

More specifically, clinicians may look for the presence of particular genetic mutations within the cells, what the DNA chromosomes physically looks like (a technique broadly called cytogenetics) and the shape of certain bone marrow blood cells (morphology).

However, although necessary, bone marrow biopsies have many downsides. Most notably the stress and physical discomfort to the patient, for which some patients require sedation. It is an invasive procedure which therefore always carries a risk of infection. This risk also increases in elderly patients, or those with a low or very low neutrophil (white blood cell) count. This makes frequent sampling problematic which means patients may not be followed as closely as clinicians would like. Overall, for many patients, regular biopsies are yet another 'painful' and inconvenient aspect of living with MDS.

Peripheral Blood Sample: An alternative to biopsy

An easier alternative to a biopsy would be a peripheral blood (PB) sample (i.e. the blood already circulating in the body, which is produced in the bone marrow).

Until recently, it had not been conclusively shown in a large scale study that PB could be used to obtain similar results as a bone marrow biopsy. Also, the commonly used testing technique, called metaphase cytogenetics, does not work very well for PB samples. Therefore until now, there has been little momentum in adopting a PB sampling as standard practice.

However recent research by a group at Kings College London and the Hospital may change that (A M Mohamedali et al). Their research has demonstrated that peripheral blood samples are an equally accurate and reliable source for monitoring the genetic mutations in bone marrow derived blood cells, and hence for monitoring the disease status of a patient (please see below for full publication details).

The research group looked for the presence of various genetic abnormalities known to be frequently associated with MDS in both bone marrow samples and PB samples, and compared the results against each other.

In order to do this, they used two specific testing methods which do work for PB samples. The first is a technique called SNP – Array karyotyping (a method used to identify changes to the number of DNA strands in a cell, a feature commonly observed in MDS). The second technique used was next-generation sequencing technology (NGS) to look at over 20 genes known to harbour mutations in up to 80% of MDS patients. They found that if a gene mutation or changes to the number of DNA strands could be detected in a bone marrow biopsy sample, it could also be detected in the PB sample of the same patient. Overall, they found that the same results could be obtained for both bone marrow biopsy and PB samples using these techniques (there was a 98% concordance in results, which is extremely high).

These are very promising results which demonstrated proof of concept that PB can be used as a substitute for bone marrow biopsies. The authors of the publication recommend the use of PB for follow-up checks and believe that PB sampling has many distinct benefits over bone marrow sampling.

The most obvious being the fact that the method is less invasive and virtually pain free, with little or no risk of infection. This allows for more frequent check-ups which in turn enables closer disease monitoring for better outcomes. The procedure is also quicker and easier to perform than a biopsy, and as no sedation is required, patients are also able to leave immediately with no recovery time required.

Aside from patient benefits there are also important advantages for hospitals too. The procedure is easier and quicker to carry out than a bone marrow biopsy, therefore does not require specialist staff and cuts down on procedure time. In some cases it may even free up hospital bed time and offer cost savings.

Additionally, once the PB sample is taken, it can be analysed relatively easily using the two testing techniques described by the research group. Both the SNP – Array karyotyping and 21st century sequencing techniques were semi-automated, reliable and provided robust results, making it attractive for hospital diagnostic labs to implement.

Although a bone marrow biopsy will always be essential for initial diagnosis, finding easier, less painful, yet still accurate and reliable ways to monitor MDS patients represents a major improvement. PB sampling could spare a large population of patients the need for repeat bone marrow biopsies, making the burden of their disease a little lighter, and allowing clinicians to follow patients more closely through more regular checks.

MDS UK – Note to patients

If you are not yet offered the choice of peripheral blood (PB) sampling during routine check-ups and would like more information about its use, please contact MDS UK. This is a fairly recent technology, therefore if your haematologist has not yet started using it please hand a copy of this article to him/her. We would be happy to provide more information directly to you and/or your haematology consultant.

Clinician and Researchers Quotes

This research has provided us with the very important information that the genetic abnormalities found in the bone marrow of MDS patients are also detected in the blood. We already know that many patients acquire new genetic abnormalities during disease progression and it would therefore be possible to monitor for this on a blood sample. At present the main limiting factors for adopting this approach are the cost of these technologies as well as the complexity of analysing the data produced. The price will however fall over time and we will continue to simplify the data analysis process meaning that this has real potential for the future management of MDS patients. Unfortunately I don’t think this will replace the need for a bone marrow biopsy as this remains critical in confirming disease progression however it may allow us to detect changes early and determine when this procedure should be performed. Further research will be needed to find out if this can improve the overall management and outcome in MDS patients.

From a clinical perspective, this study is the first of its kind to demonstrate the potential use of 21st century technologies in improving the management and treatment of human diseases, especially in a disease like MDS where the majority of the patients are of old age (> 70 years). This study has clearly shown that the genetic analysis that is usually performed on bone marrow biopsy can also be reliably done on peripheral blood, thus potentially eliminating the need for repeated painful and expensive bone marrow aspirations for disease monitoring. That being said, further larger studies involving multiple centres are needed to verify these results before being introduced into routine clinical practice. Although there are challenges that need to be addressed including the cost and the data management as well as interpretation of the results, however, this technological advancement has great potential for the clinical management of MDS patients and will also help in early intervention where disease progression is suspected.

Syed Mian, PhD, Research Associate (one of the authors of this research paper) Department of Haemato-Oncology, King’s College London

Currently only a handful of specialist laboratories are equipped to perform SNP-Array karyotyping or next generation sequencing mutation analysis in MDS. The number of centres tends to be small because these types of analysis are highly specialised, require the use of expensive, dedicated equipment and require highly skilled and experienced staff. These laboratories tend to be within specialised Haematological Malignancy Diagnostic Centers such as the service in Leeds Teaching Hospitals NHS Trust and my laboratory within King’s College Hospital London. The cost of these investigations is relatively high, however the amount of genetic information obtained using these methods is much greater and results in improved certainty of diagnosis. Some of these genetic findings are also useful for informing clinicians and patients about the likely course of the disease and can also influence treatment options in a way that the conventional methods may not. Here at King’s College Hospital we have been performing this next generation sequencing mutation analysis and SNP-array karyotyping in MDS for several years. We have performed analysis on hundreds of samples and these analyses are now available as diagnostic tests. Access to these analyses make replacement of some bone marrow biopsy samples with blood a reality for our patients.

Our study was designed primarily with the patient benefit in mind. Being a tertiary referral centre for MDS, there was a clear need to improve on existing methods in aiding patient diagnosis and enable frequent follow up of patients. The data is an extension of our earlier publication in the journal Blood (2013) and confirms the very high concordance of the genetic information obtained from the bone marrow and peripheral blood. I am delighted that MDS UK has taken the initiative to disseminate this information to the community so that patients may benefit from cutting edge research tools to help and with their MDS journey

In a joint letter to the Department of Health, Anthony Nolan Trust and some of leading names in British medicine have rallied against the new guidance, issued this month.

‘Without a second transplant, the small percentage of patients considered suitable for one will die of their underlying disease,’ said Professor David Marks, former president of the British Society of Bone Marrow Transplantation and one of 18,000 signatories, including dozens of specialists in blood disease, to a letter handed last week to Health Secretary Jeremy Hunt.

The treatment, which offers the best chance of survival to these patients, is given routinely in the US and Europe, and Prof Marks, director of the Bristol Bone Marrow Transplant Unit, says: ‘I know of no other first-world country where people with a 30 per cent chance of a cure are denied a transplant.’

Before 2013, patients in the UK eligible for a second transplant received one, but in that year NHS England (NHSE) began evaluating whether repeat transplants should continue to be funded.

For the past three years, doctors have had to submit individual funding requests. Some have been turned down, and NHSE has since declared second transplants ‘not currently affordable’.

Henny Braund, chief executive officer of British transplant charity the Anthony Nolan Trust, which co-ordinated the petition and is asking the public to write to their MPs, says: ‘This is a step backwards for patients.

How to be an Anthony Nolan bone marrow donor

Your animated guide to becoming a bone marrow donor. Steve Coogan narrates. Find out everything you ever wanted to know about donation, from joining the register to what happens if you are a match.

Calum's stem cell donation for Anthony Nolan

A transplant costs between £50,000 and £120,000, but the cost of caring for one patient refused a transplant in the past few years was £130,000 for the year they survived, and for another patient who survived three years it was £160,000.

‘Of those who do get a second transplant, one in three survive at least five years, and many are young people leading fulfilling lives and making a contribution to society. Denying those patients a chance of life amounts to a death sentence.’

One of those presenting the protest petition last week was acute myeloid leukaemia sufferer Emily Wellfare, who was told in December she would die if she did not get a second transplant.

She said: ‘My consultant at the Royal Marsden Hospital told me it would be the only thing that could save my life. 'He mentioned he would have to apply for funding, and it never occurred to me it could be refused.’

In February the 25-year-old from Eastbourne was given the shocking news that the application had been refused.

‘My doctors said the hospital was going to give me the transplant anyway. I am so grateful they fought so hard for me, but I want to know why the NHS thought my life wasn’t worth fighting for,’ added Emily.

She was in the second year of a law degree course when she started suffering from incessant coughs and colds in early 2012. In April that year she was diagnosed with acute myeloid leukaemia.

After chemotherapy, Emily spent a year in remission before discovering at her routine one-year check-up that her leukaemia had returned.

She was told she would need a transplant of stem cells – a procedure once called a bone-marrow transplant.

Cells, taken from a donor, grow in the bloodstream and make healthy blood cells to replace a patients’ own damaged ones.

Emily had her first transplant in February 2014, following three more rounds of chemotherapy.

After nearly two further years in remission, it was discovered at the end of last year that Emily’s cancer had returned again.

Since having her second transplant on March 14, Emily has been well but has to take steroids, antibacterial and anti-fungal drugs and immuno-suppressants.

Emily was luckier than another patient, a 21-year-old from Nottingham, who was rejected for a second transplant.

Dr Jenny Byrne, honorary consultant haematologist at the city’s university hospital trust, said: ‘It was extremely disappointing and frustrating, given that we have plenty of patients in Nottingham who have had second transplants and are long-term survivors, fit and well and cured.’

A spokesman for NHS England said: ‘The procedure isn’t being funded this year because its low clinical benefit and high cost meant that it compared poorly with other new treatments which have been considered for funding this year.’

He added that individual funding requests would be considered where exceptional need and benefit could be provided, and the policy would be reconsidered in 2017.

However, Dr Byrne said: ‘It is not true to say that individual funding requests for transplants are getting consideration because they are routinely screened out by administrators who claim the cases are not sufficiently unique, and it is impossible to demonstrate the level of exceptionality that is called for.’

A Department of Health spokesperson said: ‘Funding decisions around treatments such as these are rightly for NHS England. We have referred the decision to them.’

The Cancer Drug Fund (CDF)

de-listing further drugs

Many blood cancer patients are about to face more severe issues of access to treatment, following the announcement that the CDF is de-listing further drugs in order to balance its budget.

Twelve of these are blood cancer drugs.
None of the cuts affect MDS patients, but the principle is at stake – and the future funding of all cancer drugs for all cancer patients.

The Blood Cancers Alliance, an informal group of all blood cancer charities, has now written to David Cameron and Jeremy Hunt to urge them to find a suitable solution to this wholly unacceptable situation.The open letter is copied here and will be published in the Times newspaper today Wedn 4th Nov 2015.Please share it widely, including your MP.
This letter is also appearing on all websites of the Blood Cancers Alliance members.

Open letter to Rt Hon David Cameron MP and Rt Hon Jeremy Hunt MP:

Blood cancer charities urge Government for Cancer Drugs Fund solution

As an alliance of blood cancer charities, and on behalf of the 27,000 blood cancer patients and their families who have signed the petition against the delisting of life-saving drugs from the Cancer Drugs Fund (CDF), we are writing to express our concern regarding the Government’s failure on the CDF.

The Government is aware of the flawed nature of the CDF, but due to politics, has let it progress to the situation where clinically effective treatments are today being removed without a long-term solution to access.

The CDF is majorly overspent and no data has been collected on whether or not any CDF-funded treatments have actually worked. As a result of these failures 12 blood cancer drug indications, previously deemed clinically effective, are set to be removed from the Fund with no guarantee of future access.

While the CDF has improved access to cancer drugs not routinely available in NHS England, it was always intended to be a temporary solution while a long-term pricing mechanism was worked out. The proposed consultation on the CDF has taken too long to materialise, and as the new CDF system is set to be in place from April 2016, the time is rapidly decreasing for stakeholders to shape a system that is fit for purpose.

Through the revised CDF and Accelerated Access Review, there is a real opportunity to put patients at the heart of the system and ensure they are able to access the most innovative medicines. This is an opportunity that the Government can no longer afford to miss.

“On 1 April 2013, NHS England took on responsibility for the operational management of the Cancer Drugs Fund (CDF). The NHS spends approximately £1.3 billion annually on the provision of cancer drugs within routine commissioning. The CDF was established as an additional funding source to this.

The CDF has provided an additional £200m each year since then to enable patients to access drugs that would not otherwise have been routinely available from the NHS. NHS England recently pledged an additional £160m over the next two years to strengthen the fund. It was established in 2010 and will run until the end of March 2016.

There is a single, national list of drugs and indications that the CDF will routinely fund and standard operating procedures for administration of the fund.”

2015 MDS World Awareness Day: Video Stories

25/10/2015Some of our true champions for MDS awareness have shared their stories with us. Watch their video clips and share them in support of MDS World Awareness Day 2015!

Emma Paine: What happens when you need a second transplant?

Emma is an MDS patient. She had a first stem cell transplant in 2011 – and recovered well. Until now.

She is currently in hospital having chemotherapy and urgently needs a second transplant.
BUT – funding for second transplants is being revised in the UK – due to “cost-effectiveness” issues.
18 months ago, Emma would have received her potentially life-saving transplant without a problem.
Given the current NHS financial crisis, and new clinical data on the success of 2nd transplants after a relapse, the Dept of Health has tightened the rules relating to this procedure – and hospitals all over the UK need to seek special funding requests for those patients (IFR=Individual Funding Requests)

Olivia, Tilly and Ellis Hepburn make an appeal

Olivia, Tilly and Ellis, MDS super heroes and children of Lisa and Gavin Hepburn, are making an appeal for people to donate blood and stem cells to save lives of people like their Dad, who has MDS.

Lisa told us: “Today is MDS Awareness Day. Three years ago we hadn’t even heard of this disease. That’s why today is so important to make people aware. We are so grateful to the person that gave Gavin his donated bone marrow, and to the endless people that donate blood. To everyone that has helped, supported and been a shoulder to cry on these past years we are so grateful to you too…”

Their wonderful clip has already been viewed over 12,000 times on Facebook. Please keep sharing it! Thank you – on behalf of the entire MDS community.

Today is MDS Awareness Day. Three years ago we hadn’t even heard of this disease. That’s why today is so important to make people aware . We are so grateful to the person that gave Gavin his donated Bone Marrow, and to the endless people that donate blood. To everyone that has helped, supported and been a shoulder to cry on these past years we are so grateful to you too …

5 ways you can help to raise awareness about MDS

We are asking all patients, families, clinical staff, supporters to post photos or short video clips of themselves on all social media platforms, to raise awareness of this rare blood cancer MYELODYSPLASTIC SYNDROME.

All our colleagues and friends worldwide will be doing the same and will post messages online on the 25th October. See our Facebook page for more clips, and photos and the MDS Alliance website for links to all international groups marking MDS Day.

1. Record and share a video clip. You can use the message below as an example:

Hello – my name is ……. I am an MDS patient. (or I am an MDS Supporter – son/daughter/spouse/brother/sister/parent/friend/colleague/neighbour of an MDS patient). Please share my message in support of the rare blood cancer MyeloDysplastic Syndromes – and the MDS World Awareness Day on 25th October. Please support the MDS UK Patient Support Group and the international MDS Alliance. Help to register more stem cell donors and blood donors. Be a star. Thank you.

2. Share these messages of MDS patients and supporters with your family and friends

It’s #MDSWorldAwarenessDay tomorrow. Please click on my video to see how you can help! #MDSUKPatientSupportGroup

100 Q&A book on MDS – revised edition

We now have a brand new revised edition of the really helpful 100 Questions & Answers book on MDS available from our office – free of charge to patients.

Some of you may have been given the initial 2008 edition, which we distributed a few years ago – and was extremely popular amongst our members.

For those of you who don’t know it – this is a patient friendly book on MDS, providing detailed information on several aspects of MDS, including chapters on :

• What is MDS?
• Is it a cancer?
• How is MDS diagnosed?
• What’s the best treatment for me?

This brand new version was published in July 2015, and contains all up to date information, including the revised MDS prognostic score – IPSS-R.

This revised version is fully endorsed by MDS experts, the MDS Foundation, the MDS Alliance, and or course MDS UK.

Please note, as this is a publication written for a USA patient audience, some of the drugs may not yet be available in the UK/Europe and some of the measurements for blood counts may differ. Please contact us if you if you have any queries.

As previously, we will be distributing the book at all of our regional and national patient meetings, and on request via our office.

Display copies for your hospital

We will provide a display copy at all major hospitals, alongside our leaflets and newsletters. Please let us know if you’d like to have a display copy for your hospital – so that more patients can take advantage of the information.

As a not for profit organisation, MDS UK was very kindly given a price discount by the publishers – Jones & Bartlett Learning – for which we are extremely grateful!
As we are offering the booklet for free to patients, we still have to raise some funds to purchase further copies.
If you are able to help make this booklet available to more patients, please consider donating towards this project via this specific Virgin Money Giving page:

London School of Economics Survey about how quality of life is measured and reported in Health Technology Assessments (HTA)

28/05/15

Another survey for patients and carers to take part.

HTA organisations, such as for example NICE (National Institute for Clinical Health and Excellence) mostly use a questionnaire called the EQ-5D to measure the impact of an illness. From our experience with MDS patients, we have found this EQ-5D questionnaire inadequate to reflect the various issues faced by our group of patients. This survey is an opportunity to test and evaluate this widely used questionnaire.
We therefore welcome this survey and project – and encourage you to take part – both patients and caregivers/carers.
Thank you.

Here is the official invitation, background and links to the survey:

The London School of Economics (LSE) is leading an EU-funded research project, Advance-HTA, looking at aspects of health technology assessment (a method used to determine where and how new treatments should be introduced into a national healthcare system). The goal is to work with a range of stakeholders to explore how the methods used in HTA can be improved to better meet the needs of both patients and modern healthcare systems. There are 13 institutions involved in the research from a number of EU countries and North America.

One area of particular interest is the way in which patients’ experience in quality of life is measured. Currently, a tool called EQ-5D-5L is used but there is considerable discussion about how well this reflects the aspects of health-related quality of life that matter to people living with an illness.

We are asking patients with a range of illnesses, from a number of countries, to complete a short questionnaire aimed at finding out ‘Does EQ-5D-5L accurately and reliably reflect the aspects of their health that matter most to patients?’. This is important because it will help ensure that the things that matter to patients are at the heart of future decisions about access to medicines.

FOR CAREGIVERS
Another version of the questionnaire has been developed to capture the responses of the caregivers who are providing support and care for patients. If you know any family members and friends who are caring for a patient, please provide them with the following link to complete the questionnaire: https://lse.qualtrics.com/SE/?SID=SV_9sM3VwpFLDT1xVH

Each questionnaire must be completed in one sitting and all responses are anonymous.

If you are involved in a patient organisation, please consider posting the links to the surveys on your organisation’s website. If you know other patient organisations in Europe, please do forward them the links. The more completed questionnaires we receive, the more reliable the results will be.

Thank you very much for taking the time to complete the questionnaire and support this important piece of research. More information about the project and the institutions involved can be found athttp://www.advance-hta.eu.